Clinical trials in ALD that are recruiting or otherwise active, compiled from registrations on ClinicalTrials.gov.
Before reading If you are considering participation, discuss it with your treating clinicians first. Every trial has strict eligibility criteria — age, phenotype, disease stage, prior treatment — and the summaries here cannot tell you whether you qualify.
No trial has a site in Korea
Of the 74 ALD-related studies registered on ClinicalTrials.gov, not one has a study site in Korea.
This is more than an inconvenience. It means Korean patients will reach new treatments last. For a sponsor to consider Korea as a trial location, there has to be data on how many patients there are and what condition they are in — and at present that does not exist.
Therapeutic trials
Leriglitazone in adult cerebral ALD (CALYX)
A phase 3 trial of the efficacy and safety of leriglitazone in adult men with cerebral disease. In paediatric cerebral ALD the drug has reached a positive regulatory opinion in Europe; this trial addresses adults.
ACTIVE_NOT_RECRUITING — running, not currently enrolling
Sponsor: Minoryx Therapeutics · Started July 2023
→ NCT05819866
Dimethyl fumarate in adrenomyeloneuropathy
A phase 2/3 trial in AMN. Dimethyl fumarate, used in multiple sclerosis, targets oxidative stress and neuroinflammation.
ACTIVE_NOT_RECRUITING
Principal investigator: Aurora Pujol (Spain) · Started April 2024
→ NCT06513533
Treating leg symptoms in women with X-linked ALD
A phase 4 trial addressing leg symptoms in women with ALD — an unusual study in that it is a therapeutic trial in female patients only.
ACTIVE_NOT_RECRUITING
Massachusetts General Hospital · Started April 2023
→ NCT05003648
Intrathecal and intravenous lentiviral gene therapy
Lentiviral gene therapy delivered both intrathecally and intravenously.
RECRUITING
Shenzhen Geno-Immune Medical Institute (China) · Started August 2025
→ NCT03727555
Transplantation trials
Reduced intensity conditioning for non-malignant disorders
A phase 2 trial of reduced-intensity conditioning in umbilical cord blood, bone marrow and peripheral blood stem cell transplantation, including ALD among non-malignant indications.
RECRUITING · Paul Szabolcs (University of Pittsburgh) · Started 2014
→ NCT01962415
Cord blood transplant with intrathecal cord blood–derived oligodendrocyte-like cells
A phase 1 trial in inherited metabolic diseases, combining cord blood transplantation with intrathecal administration of cord blood–derived cells.
RECRUITING · Joanne Kurtzberg (Duke University) · Started 2014
→ NCT02254863
Allogeneic HCT for metabolic disorders and severe osteopetrosis (MT2013-31)
ACTIVE_NOT_RECRUITING · University of Minnesota · Started 2014
→ NCT02171104
Symptom management
Epidural spinal cord stimulation for lower-limb impairment in AMN
RECRUITING · Third Military Medical University (China) · Started February 2025
→ NCT06796920
Home exercise for individuals with neurodegenerative disease
RECRUITING · Kennedy Krieger Institute · Started 2023
→ NCT05443906
Registries and observational studies
These are not treatment studies, but taking part contributes to bringing the next treatment forward. Data on how many patients there are and what course they follow is what allows new trials to be designed.
| Study | Sponsor | Status |
|---|---|---|
| ALD National Registry Study | University of Minnesota | RECRUITING |
| Registry of X-linked ALD | Beijing Tiantan Hospital (China) | RECRUITING |
| Quality of life in women with X-linked ALD | Leipzig University (Germany) | RECRUITING |
| Disease progression in women with X-linked ALD | Minoryx Therapeutics | RECRUITING |
| Myelin Disorders Biorepository Project | Children’s Hospital of Philadelphia | RECRUITING |
| Longitudinal study of ultra-rare metabolic and degenerative neurological disease | Istituto Neurologico Carlo Besta (Italy) | RECRUITING |
| Macrophage activation patterns in X-linked ALD | AP-HP (France) | RECRUITING |
| Prognostic biomarker using brain diffusion MRI | AP-HP (France) | NOT_YET_RECRUITING |
| Follow-up of patients treated with elivaldogene autotemcel | Genetix Biotherapeutics | RECRUITING |
| Long-term follow-up after lentiviral gene therapy | Genetix Biotherapeutics | ACTIVE_NOT_RECRUITING |
| Early Check: expanded newborn screening | RTI International | ACTIVE_NOT_RECRUITING |
Searching directly
This page is updated periodically, but the source is always more current.
→ Search ClinicalTrials.gov for “adrenoleukodystrophy”
Filtering by RECRUITING shows only studies currently open to enrolment.
What the status labels mean
| Label | Meaning |
|---|---|
RECRUITING | Currently enrolling participants |
NOT_YET_RECRUITING | Registered but enrolment has not opened |
ENROLLING_BY_INVITATION | Enrolling selected participants only |
ACTIVE_NOT_RECRUITING | Running, but enrolment has closed |
Compiled from ClinicalTrials.gov registrations as of 7 August 2026. Trial status and eligibility change frequently.
Last reviewed: 7 August 2026